Evidence mapPaperPMID 32030781Full record

GuidelineJournal of inherited metabolic disease2020

Safety of drug use in patients with a primary mitochondrial disease: An international Delphi-based consensus.

Maaike C De Vries, David A Brown, Mitchell E Allen, Laurence Bindoff, Gráinne S Gorman, Amel Karaa, Nandaki Keshavan, Costanza Lamperti, Robert McFarland, Yi Shiau Ng and 7 more

Open access · hybridAbstract readPractice Guideline
In one paragraph

Guideline in Journal of inherited metabolic disease, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
33citing papers in PubMed, 1 pooled it
3.6field-weighted citation impact, top 6% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

33 citing papers in PubMed, 1 synthesis or guideline pooled it, 65 citations in OpenAlex.

  1. Guideline
  2. Drug safety in patients with mitochondrial disease: an observational cohort study.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2026
    Observational
  3. Prevalence and treatment of mitochondrial diabetes in Southwest Finland.Journal of diabetes and metabolic disorders · 2026
    Article
  4. Article
  5. Article
  6. Article
  7. Review
  8. Review
  9. Review
  10. Review
  11. Reply.AJNR. American journal of neuroradiology · 2024
    Article
  12. Article
  13. Review
  14. Article
  15. A clinical approach to diagnosis and management of mitochondrial myopathies.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024
    Review
  16. Red Flags in Primary Mitochondrial Diseases: What Should We Recognize?International journal of molecular sciences · 2023
    Review
  17. Article
  18. Article
  19. Article
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

17 authors at 10 institutions in 6 countries.

Maaike C De VriesRadboudumc Amalia Children's Hospital, Radboud Center for Mitochondrial Medicine, Nijmegen, The Netherlands.ORCID 0000-0003-4316-5481
David A BrownDepartment of Human Nutrition, Foods, and Exercise and the Virginia Tech Center for Drug Discovery, Virginia Tech, Blacksburg, Virginia.
Mitchell E AllenDepartment of Human Nutrition, Foods, and Exercise and the Virginia Tech Center for Drug Discovery, Virginia Tech, Blacksburg, Virginia.
Laurence BindoffDepartment of Clinical Medicine, University of Bergen, Bergen, Norway.
Gráinne S GormanWellcome Centre for Mitochondrial Research, Institute of Neuroscience, Newcastle University, Newcastle upon Tyne, UK.
Amel KaraaGenetics Unit, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Nandaki KeshavanMitochondrial Research Group, UCL Great Ormond Street Institute of Child Health, London, UK.
Costanza LampertiUnit of Medical Genetics and Neurogenetics, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
Robert McFarlandWellcome Centre for Mitochondrial Research, Institute of Neuroscience, Newcastle University, Newcastle upon Tyne, UK.
Yi Shiau NgWellcome Centre for Mitochondrial Research, Institute of Neuroscience, Newcastle University, Newcastle upon Tyne, UK.
Mar O'CallaghanDepartment of Neurology, Metabolic Unit, Hospital Sant Joan de Déu, Barcelona, Spain.
Robert D S PitceathlyDepartment of Neuromuscular Diseases, UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery, London, UK.
Shamima RahmanMitochondrial Research Group, UCL Great Ormond Street Institute of Child Health, London, UK.
Frans G M RusselDepartment of Pharmacology and Toxicology, Radboud Institute for Molecular Life Sciences, Radboud Center for Mitochondrial Medicine, Radboudumc, Nijmegen, The Netherlands.
Kristin N VarhaugDepartment of Clinical Medicine, University of Bergen, Bergen, Norway.
Tom J J SchirrisDepartment of Pharmacology and Toxicology, Radboud Institute for Molecular Life Sciences, Radboud Center for Mitochondrial Medicine, Radboudumc, Nijmegen, The Netherlands.
Michelangelo MancusoDepartment of Clinical and Experimental Medicine, Neurological Institute, University of Pisa, Pisa, Italy.
Newcastle upon Tyne Hospitals NHS Foundation Trust · GBRadboud University Nijmegen · NLGreat Ormond Street Hospital for Children NHS Foundation Trust · GBHaukeland University Hospital · NOVirginia Tech · USFondazione IRCCS Istituto Neurologico Carlo Besta · ITHarvard University · USHospital Sant Joan de Déu Barcelona · ESNational Hospital for Neurology and Neurosurgery · GBUniversity of Pisa · IT

Funding

Biotechnology and Biological Sciences Research CouncilDepartment of Health CL-2016-01-003Medical Research Council 13/NE/0326Medical Research Council L016354Medical Research Council MR/S002065/1NHLBI NIH HHS R01 HL123647NHLBI NIH HHS R56 HL123647Wellcome Trust 203105/Z/16/ZWellcome Trust 204709/Z/16/Z
6 · The paper itself

Abstract

Clinical guidance is often sought when prescribing drugs for patients with primary mitochondrial disease. Theoretical considerations concerning drug safety in patients with mitochondrial disease may lead to unnecessary withholding of a drug in a situation of clinical need. The aim of this study was to develop consensus on safe medication use in patients with a primary mitochondrial disease. A panel of 16 experts in mitochondrial medicine, pharmacology, and basic science from six different countries was established. A modified Delphi technique was used to allow the panellists to consider draft recommendations anonymously in two Delphi rounds with predetermined levels of agreement. This process was supported by a review of the available literature and a consensus conference that included the panellists and representatives of patient advocacy groups. A high level of consensus was reached regarding the safety of all 46 reviewed drugs, with the knowledge that the risk of adverse events is influenced both by individual patient risk factors and choice of drug or drug class. This paper details the consensus guidelines of an expert panel and provides an important update of previously established guidelines in safe medication use in patients with primary mitochondrial disease. Specific drugs, drug groups, and clinical or genetic conditions are described separately as they require special attention. It is important to emphasise that consensus-based information is useful to provide guidance, but that decisions related to drug prescribing should always be tailored to the specific needs and risks of each individual patient. We aim to present what is current knowledge and plan to update this regularly both to include new drugs and to review those currently included.

Indexed as

Drug-Related Side Effects and Adverse ReactionsMitochondriaMitochondrial DiseasesPharmaceutical PreparationsConsensusDelphi TechniqueDrug DesignHumansInternationalityToxicity TestsPharmaceutical Preparationsdrugsin vitro studiesin vivo studiesmitochondrial diseasesmitochondrial toxicitysafety

Identifiers

PMID32030781
PMCPMC7383489
OpenAlexW3004722903

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.